Selective Recognition of Folded RNA by Small Oligomers

新的药物靶点对于寻找新的和更有效的治疗方法来减轻人类疾病是很重要的。目前，作为药物开发的潜在靶标，RNA和相关RNA结合蛋白的利用明显不足：很可能是因为缺乏关于如何设计靶向这些结构的分子的基本知识。在我们的研究中，我们正在开发一种小分子来干扰核衣壳蛋白7，NCp7及其与病毒HIV RNA的关联。为了使HIV正常成熟，NCp7必须与HIV RNA结合并保护其不被降解。干扰这一过程是一种阻止艾滋病毒发展的策略，可能成为艾滋病毒感染者的一种有价值的新治疗方法。我们继续开发我们的小分子，我们正在从事必要的转化工作，将我们的分子推向临床试验。在过去的一年里，我们已经确定这种分子是口服生物可利用的，对大鼠无毒。对该分子安全性的进一步研究将随着对其作用机制的进一步研究而继续进行。

New drug targets are important to study to find new and more effective therapies to alleviate human disease. Currently, RNA and associated RNA-binding proteins are significantly underutilized as a potential target for drug development: most likely because there exists a lack of basic knowledge about how one should design a molecule to target these structures. In our research, we are developing a small molecule to interfere with nucleocapid protein 7, NCp7, and its association with viral HIV RNA. In order for HIV to mature properly, NCp7 must bind to HIV RNA and protect it from degradation. Interfering with this process is a strategy to halt with HIV's progression and could become a valuable new treatment for HIV infected individuals. We have continued to develop our small molecule and are we are engaged in the necessary translational work to move our molecule toward a clinical trial. Over the past year, we have determined that the molecule is orally bioavailable and not toxic to rats. Further studies on the safety of this molecule will continue along with more investigations into the mechanism of action.